The clinical phase 1 study of the T regulatory (Treg) cell product CK0801 in nine patients with bone marrow failure syndromes reported by Kadia et al. in NEJM Evidence fits well into the current immunotherapy era. These cells, brought to the forefront by Shimon Sakaguchi two decades ago, constitute a subpopulation of CD4+ T cells characterized by the surface expression of the alpha chain of the interleukin-2 receptor, CD25, the inhibitory molecule CTLA-4, and the intracellular transcription factor FoxP3. They control, under healthy conditions and through several mechanisms, expansion and function of other immune cells, such as effector T cells and natural killer cells. Defective Treg functions lead to autoimmunity but also contribute to other diseases in which the immune system is involved. In an editorial, Carole Seguin-Devaux, PhD, and Jacques Zimmer, MD, PhD, review the safety and preliminary efficacy results of CK0801. Read the full editorial: https://eviden.cc/4bgUGk8 𝗙𝗨𝗥𝗧𝗛𝗘𝗥 𝗥𝗘𝗔𝗗𝗜𝗡𝗚 Original Article by Tapan Kadia, MD, simrit parmar, MD, MSCI, et al.: Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes https://eviden.cc/4bkpyjR #ClincialTrials #MedicalResearch
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